Skytrofa® (lonapegsomatropin-tcgd) is now available for the treatment of patients 1 year of age and older weighing at least 11.5kg with growth failure due to inadequate secretion of endogenous growth hormone.
Skytrofa is a long-acting prodrug of the human growth hormone, somatropin, produced by recombinant DNA technology using E. coli. The approval was based on data from the multicenter, randomized, open-label, active-controlled, parallel-group phase 3 heiGHt trial (ClinicalTrials.gov Identifier: NCT02781727), which compared the efficacy and safety of once-weekly lonapegsomatropin-tcgd to daily somatropin in 161 treatment-naïve, prepubertal patients with growth hormone deficiency.
Findings from the study showed that lonapegsomatropin-tcgd met the noninferiority criteria for the primary endpoint demonstrating a greater annualized height velocity at week 52 compared with somatropin. The most common adverse reactions reported were viral infection, pyrexia, cough, nausea/vomiting, hemorrhage, diarrhea, abdominal pain, and arthralgia and arthritis.
“Skytrofa offers patients, caregivers, and physicians the potential to replace daily somatropin injections that have been the standard of care for more than 30 years,” said Jan Mikkelsen, Ascendis Pharma’s President and CEO. “As the first and only FDA-approved once-weekly therapy for pediatric growth hormone deficiency, Skytrofa represents one of the most important innovations for these patients in decades.”
Skytrofa is supplied as a single-dose, dual-chamber, prefilled cartridge containing lonapegsomatropin-tcgd and diluent for subcutaneous injection in the following strengths: 3mg, 3.6mg, 4.3mg, 5.2mg, 6.3mg, 7.6mg, 9.1mg, 11mg and 13.3mg.
The cartridges are for use only with the Skytrofa Auto-Injector, which is packaged separately. Selection of the appropriate cartridge is based on prescribed dose and the patient’s body weight.
The Company is also offering a patient support program to assist eligible patients.
- Ascendis Pharma A/S announces US commercial launch of Skytrofa® (lonapegsomatropin-tcgd), the first and only FDA approved once-weekly treatment for pediatric growth hormone deficiency. News release. Ascendis Pharma A/S. Accessed October 15, 2021. https://www.globenewswire.com/news-release/2021/10/15/2314984/0/en/Ascendis-Pharma-A-S-Announces-U-S-Commercial-Launch-of-SKYTROFA-Lonapegsomatropin-tcgd-the-First-and-Only-FDA-Approved-Once-Weekly-Treatment-for-Pediatric-Growth-Hormone-Deficiency.html.
- Skytrofa. Package insert. Ascendis Pharma; 2021. Accessed October 15, 2021. https://ascendispharma.us/products/pi/skytrofa/skytrofa_pi.pdf.